Policymakers Seek New Ways to Streamline R&D and Approval

One prominent response to mounting outrage over the high cost of new biotech medicines is to find ways to make drug development and clinical research more efficient and informative. Congressional leaders have launched a major initiative to revise laws and regulatory policies to speed new cures to patients. Biopharmaceutical companies are collaborating with public and private entities in hopes that sharing data and expertise will produce new research tools and speed the identification of potentially effective drugs. And healthcare analysts propose new approaches to lower the high risks of biomedical discovery by reducing the $1 billion cost of bringing a new drug to market.

One response from industry is to negotiate billion-dollar deals to strengthen drug development pipelines by focusing more on treatment areas where they have greater expertise. The latest wave of mega-mergers and asset exchanges are expected to have notable impacts on pharma R&D programs and clinical trial operations as re-shaped companies take a hard look at research portfolios to cull out less promising programs.

More breakthroughs

FDA is promoting its broad range of strategies to speed product development and review. Commissioner Margaret Hamburg acknowledged in a keynote speech at the April annual meeting of the Food and Drug Law Institute (FDLI) that FDA is accused of having only two speeds of approval—"too fast and too slow"—which either delay approval of vital new therapies, or sacrifice standards and consistency for speedy reviews.

Such concerns are being offset by the success of FDA's breakthrough therapy designation, authorized by Congress in 2012. Janet Woodcock, director of the Center for Drug Evaluation and Research (CDER), agreed that even though FDA has had a number of programs for accelerating reviews and patient access to new drugs, the breakthrough designation is a "game changer" for addressing serious, life threatening conditions. And sponsors are enthusiastic: CDER received 144 requests for breakthrough designation through April 4, granted 40 and denied 70, Woodcock reported, noting that the requests are not just for cancer drugs, but also for antivirals and other diseases. Last year, nearly half of all new drugs approved took advantage of an expedited review pathway, and several were cleared in less than six months.

FDA's breakthrough policy and other mechanisms for speeding up review and approval of important new drugs should be extended to medicines expected to substantially reduce spending, according to a recent study by the Rand Corp. Its report on "Redirecting Innovation in U.S. Health Care" proposes new incentives and strategies to reduce the cost of developing high-value medical products as a way to rein in medical spending. Expedited approval based on the potential to cut costs would require new legislation to expand FDA's mission beyond safety and efficacy, as well as methods for determining how and when a new product truly has economic benefits.

Congress weighs in

These and other possibilities are being examined by the House Energy & Commerce Committee, which launched a "21st Century Cures" initiative last month to explore the need for new laws and regulatory reform to promote biomedical innovation. Committee chairman Fred Upton (R-Mich.) and leading Democrat Diana DeGette (D-Col.) emphasize the bipartisan nature of the undertaking and collaboration with FDA, the National Institutes of Health (NIH), and other agencies in seeking ways to streamline product development.

An initial white paper questions whether randomized, blinded clinical trials that compare the effect of a drug over time on large numbers of patients is always the best approach. The "timelines, size, failure rates, and costs of conducting trials are at all-time highs," the committee states. While the policy-makers acknowledge FDA involvement in numerous partnerships to develop new biomarkers, modernize clinical trial operations, and promote innovative research approaches, they emphasize the need to explore new ways to improve the process, including whether FDA is structured and managed so that it can rapidly incorporate innovative approaches and technologies into its review process.

The committee launched its investigation last month with a roundtable talk on the state of U.S. biomedical innovation and the potential for China and other nations to gain preeminence in the field. Panel members included Woodcock, NIH director Francis Collins, and experts from academia, think tanks, the investment community, and patient groups.

These discussions may lead to adoption of specific Congressional proposals, possibly through legislation to renew prescription drug user fees for 2017. There are bills before Congress to expand incentives for developing new antibiotics to address drug-resistant infectious diseases, and growing concern on Capitol Hill about rampant abuse of prescription painkillers and opioids. These and other issues will factor into negotiations between FDA and industry on PDUFA VI, which are slated to begin next year.